Systems for clinical trials

Inactive Publication Date: 2010-11-04
WISTA LAB LTD
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  • Abstract
  • Description
  • Claims
  • Application Information

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Benefits of technology

[0013]This unexpected finding by the present inventors thereby highlighted a new problem in dem

Problems solved by technology

1. As noted above, one surprising discovery of the present invention is that that the primary trial design assumption was false in the rember™ study, i.e. patients who were CDR-mild at baseline did not significantly

Method used

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  • Systems for clinical trials
  • Systems for clinical trials
  • Systems for clinical trials

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example embodiments

[0183]Thus in one embodiment the subject group is stratified into mild AD (CDR=1) and moderate AD (CDR=2). Another sub-group may be Mild Cognitive Impairment (MCI as defined by agreed clinical criteria or CDR=0.5).

[0184]In the MCI and mild AD sub-group the time frame may be insufficient to expect, in the light of the results described herein, decline in the psychometric outcome measure (e.g. less than 6 months).

[0185]Thus physiological outcome measures are used in addition to psychometric outcome measures. These may be in the time frame up to 6 months for mild AD (e.g. 3 to 4 months), and even longer (6-12 months for MCI). As shown herein, efficacy demonstrated by such measures can predict future efficacy using clinical-psychometric end-points.

[0186]In mild or moderate AD, dosage strengths of a putative disease-modifying treatment, which may produce limited or no apparent therapeutic efficacy over 6 months, but which produces evidence of physiological efficacy over 6 months, can be ...

example 1

Disease Severity at Baseline and Rate of Disease Progression

[0200]It has been generally recognised in the literature that severity or stage of disease is an important predictor of disease progression (reviewed recently in Schaufele, M., Bickel, H., Weyerer, S. (2002) Which factors influence cognitive decline in older adults suffering from dementing disorders? International Journal of Geriatric Psychiatry, 17:1055-1063).

[0201]However the rember™ study is the first in which CDR severity at baseline was pre-specified as a stratification covariate in the primary outcome analysis. Subjects were classified into two groups: those who were CDR-mild at baseline (including 3 [1% of total randomised] who were CDR-questionable at baseline) and those who were CDR moderate at baseline. Although CDR has been advocated previously as a staging instrument (Berg, L., Danziger, W. L., Storandt, M., Coben, L. A., Gado, M., Hughes, C. P., Knesevich, J. W., Botwinick, J. (1984) Predictive features in mild...

example 2

Cognitive Reserve

[0209]Although there is a general relationship between increased load of brain pathology and decline in cognitive function, this relationship does not explain all of the variance in the data. Attempts to explain the variability have been formulated in terms of the concepts of “brain reserve” or “cognitive reserve”, which relate to two different theoretical formulations (Stern, Y. (2002) What is cognitive reserve? Theory and research application of the reserve concept. Journal of the International Neuropsychological Society, 8:448-460). The first is the passive brain reserve model, where reserve is defined by brain size or neuronal count. It is described as passive because it is defined in terms of the amount of damage or burden an individual can withstand before clinical symptoms appear. The second, or active cognitive reserve model, suggests that the brain can compensate for pathological burden by recruiting other processes to perform tasks compromised by disease (...

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Abstract

The invention provides methods and systems for assessing the efficacy of a pharmaceutical which is putatively disease modifying of a cognitive disorder, for use in the treatment or prophylaxis of that cognitive disorder, the method comprising the steps of: (1) stratifying a subject group into at least 2 sub-groups according to a baseline indicator of likely disease progression, (2) treating members of each subject group with the pharmaceutical for a treatment time frame, (3) deriving psychometric and optionally physiological outcome measures for each treated patient group, (4) comparing the outcomes at (3) with a comparator arm of said sub-groups which is optionally a placebo or minimal efficacy comparator arm, (5) using the comparison in (4) to derive an efficacy measure for the pharmaceutical. The methods and systems of the invention address problems such as low rate of decline over the treatment time-frame of patients who have mild-disease severity at baseline and biased withdrawal, particularly in the placebo/comparator treatment arm.

Description

TECHNICAL FIELD[0001]The present invention relates generally to methods for use in assessing the efficacy of a pharmaceutical treatments for cognitive disorders, particularly in respect of physiological and psychometric outcomes for putatively disease-modifying treatments.BACKGROUND ART[0002]Clinical Trials for Disease Modifying Treatments[0003]3,7-diaminophenothiazine (DAPTZ) compounds including methylthioninium chloride (“MTC”) have previously been shown to inhibit tau protein aggregation and to disrupt the structure of PHFs, and reverse the proteolytic stability of the PHF core (see WO96 / 30766, F Hoffman-La Roche). Such compounds were disclosed for use in the treatment and prophylaxis of various diseases, including Alzheimer's Disease (AD) and Lewy body disease. The rationale for the potential efficacy of DAPTZ compounds in the treatment and prophylaxis of disorders such as AD is based on their ability to act on the primary neurofibrillary pathology of Alzheimer's disease discove...

Claims

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Application Information

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IPC IPC(8): G06Q90/00A61B5/00G06F19/00
CPCG06F19/3437G06Q99/00G06F19/363G16H10/20G16H50/50
Inventor WISCHIK, CLAUDE MICHELWISCHIK, DAMON JUDESTAFF, ROGER TODDMURRAY, ALISON DOROTHY
Owner WISTA LAB LTD
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